A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy

NCT ID: NCT05444257

Last Updated: 2025-09-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

822 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-11-08

Study Completion Date

2026-10-30

Brief Summary

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The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.

Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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VX-121/TEZ/D-IVA

Part A: Participants will receive VX-121/TEZ/D-IVA once daily for 96 weeks. Part B: Participants will receive VX-121/TEZ/D-IVA once daily for an additional 48 weeks.

Group Type EXPERIMENTAL

VX-121/TEZ/D-IVA

Intervention Type DRUG

Fixed-dose combination tablets for oral administration.

Interventions

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VX-121/TEZ/D-IVA

Fixed-dose combination tablets for oral administration.

Intervention Type DRUG

Other Intervention Names

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VX-121/VX-661/CTP-656 VX-121/VX-661/VX-561 VX-121/tezacaftor/deutivacaftor

Eligibility Criteria

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Inclusion Criteria

* Part A: Completed study drug treatment in a parent study VX20-121-102 (NCT05033080) and VX20-121-103 (NCT05076149); or had study drug interruption(s) in a parent study but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period in the parent study
* Part B: Completed study drug treatment in Part A; or had study drug interruption(s) in Part A, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of Part A

Exclusion Criteria

* History of drug intolerance in a parent study
* Pregnant or breast-feeding females at the time of enrollment in Part A
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

Banner University of Arizona Medical Center

Tucson, Arizona, United States

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University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

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Miller Children's Hospital / Long Beach Memorial

Long Beach, California, United States

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Children's Hospital Los Angeles

Los Angeles, California, United States

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Kaiser Permanente

Oakland, California, United States

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University of California Davis Medical Center

Sacramento, California, United States

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University of California San Francisco, Lung Transplant Program

San Francisco, California, United States

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Children's Hospital of Colorado

Aurora, Colorado, United States

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National Jewish Health

Denver, Colorado, United States

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University of Florida, Shands Hospital

Gainesville, Florida, United States

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Joe DiMaggio Cystic Fibrosis & Pulmonary Center

Hollywood, Florida, United States

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University of Miami Miller School of Medicine

Miami, Florida, United States

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Central Florida Pulmonary Group,PA

Orlando, Florida, United States

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Nemours Children's Specialty Care

Pensacola, Florida, United States

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Johns Hopkins All Children's Hospital Outpatient Care Center

St. Petersburg, Florida, United States

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The Emory Clinic at Chantilly

Atlanta, Georgia, United States

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Augusta University

Augusta, Georgia, United States

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St. Luke's Cystic Fibrosis Center of Idaho

Boise, Idaho, United States

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Northwestern Memorial Hospital

Chicago, Illinois, United States

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Indiana University

Indianapolis, Indiana, United States

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Riley Hospital for Children at Indiana University Health

Indianapolis, Indiana, United States

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University of Kansas Medical Center

Kansas City, Kansas, United States

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University of Kentucky

Lexington, Kentucky, United States

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Tulane Medical Center

New Orleans, Louisiana, United States

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Maine Medical Partners

Portland, Maine, United States

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Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center

Boston, Massachusetts, United States

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Boston Children's Hospital

Boston, Massachusetts, United States

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UMass Memorial Medical Center

Worcester, Massachusetts, United States

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Michigan Medicine

Ann Arbor, Michigan, United States

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Harper University Hospital

Detroit, Michigan, United States

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Spectrum Health Medical Group Adult Cystic Fibrosis Care Center

Grand Rapids, Michigan, United States

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University of Minnesota

Minneapolis, Minnesota, United States

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Childrens Hospital University of Missouri Health Sciences Center

Columbia, Missouri, United States

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The Children's Mercy Hospital

Kansas City, Missouri, United States

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Cardinal Glennon Children's Hospital - St. Louis University

St Louis, Missouri, United States

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Washington University School of Medicine / St. Louis Children's Hospital

St Louis, Missouri, United States

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Billings Clinic

Billings, Montana, United States

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Nebraska Medical Center

Omaha, Nebraska, United States

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Dartmouth Hitchcock

Manchester, New Hampshire, United States

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Morristown Medical Center

Morristown, New Jersey, United States

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Albany Medical College, Pulmonary and Critical Care Medicine

Albany, New York, United States

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CF Therapeutics Development Center of Western New York

Buffalo, New York, United States

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Long Island Jewish Medical Center

New Hyde Park, New York, United States

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Columbia University Medical Center

New York, New York, United States

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SUNY Upstate Medical University

Syracuse, New York, United States

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New York Medical College

Valhalla, New York, United States

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Atrium Health Levine Children's Hospital

Charlotte, North Carolina, United States

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Wake Forest Baptist Health

Winston-Salem, North Carolina, United States

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Akron Children's Hospital

Akron, Ohio, United States

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Nationwide Children's Hospital

Columbus, Ohio, United States

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Dayton Children's Hospital

Dayton, Ohio, United States

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ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center

Toledo, Ohio, United States

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University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, United States

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Santiago Reyes, M.D.

Oklahoma City, Oklahoma, United States

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Oregon Health & Science University

Portland, Oregon, United States

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Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

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UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

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Prisma Health Richland Campus

Columbia, South Carolina, United States

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Sanford Children's Speciality Clinic

Sioux Falls, South Dakota, United States

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University of Tennessee Medical Center

Knoxville, Tennessee, United States

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Vanderbilt University Medical Center, Nephrology Clinical Trials Center

Nashville, Tennessee, United States

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The University of Texas Southwestern Medical Center

Dallas, Texas, United States

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Cook Children's Health Care System

Fort Worth, Texas, United States

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Texas Children's Hospital - Baylor College of Medicine

Houston, Texas, United States

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University of Utah

Salt Lake City, Utah, United States

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Vermont Lung Center

Colchester, Vermont, United States

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University of Virginia Health System

Charlottesville, Virginia, United States

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University of Washington Medical Center

Seattle, Washington, United States

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Providence Pediatric Pulmonary & Cystic Fibrosis Clinic

Spokane, Washington, United States

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West Virginia University

Morgantown, West Virginia, United States

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University Hospital and UW Health Clinics

Madison, Wisconsin, United States

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Royal Prince Alfred Hospital

Camperdown, , Australia

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The Prince Charles Hospital

Chermside, , Australia

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Alfred Hospital

Melbourne, , Australia

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Institute for Respiratory Health

Nedlands, , Australia

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Telethon Kids Institute

Nedlands, , Australia

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The Royal Children's Hospital

Parkville, , Australia

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Mater Adult Hospital

South Brisbane, , Australia

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Queensland Children's Hospital

South Brisbane, , Australia

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Westmead Hospital

Westmead, , Australia

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University of Graz

Graz, , Austria

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Medizinische Universität Innsbruck

Innsbruck, , Austria

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Uniklinikum Salzburg - Universitätsklinik für Pneumologie/Lungenheilkunde

Salzburg, , Austria

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Medizinische Universität Wien

Vienna, , Austria

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Cliniques Universitaires de Bruxelles Hopital Erasme

Brussels, , Belgium

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Universitair Ziekenhuis Brussel - Campus Jette

Brussels, , Belgium

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Universitair Ziekenhuis Gent

Ghent, , Belgium

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Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

Leuven, , Belgium

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Cliniques Universitaires Saint-Luc

Woluwe-Saint-Lambert, , Belgium

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University of Calgary Medical Clinic of the Foothills Medical Centre

Calgary, , Canada

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Stollery Children's Hospital

Edmonton, , Canada

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St. Joseph's Health Care London

London, , Canada

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Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu

Montreal, , Canada

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McGill University Health Centre, Glen Site, Montreal Children's Hospital

Montreal, , Canada

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Institut Universitaire de Cardiologie et Pneumologie de Quebec - Universite Laval

Québec, , Canada

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British Columbia Children's Hospital

Vancouver, , Canada

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St. Paul's Hospital

Vancouver, , Canada

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Klinika Detskych Infekcnich Nemoci

Brno, , Czechia

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Fakultni nemocnice v Motole

Prague, , Czechia

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Aarhus University Hospital

Aarhus, , Denmark

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Juliane Marie Center, Rigshospitalet

Copenhagen, , Denmark

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Groupe Hospitaler Pellegrin, CHU De Bordeaux

Bordeaux, , France

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CHU Lyon - Hopital Femme Mere-Enfant

Bron, , France

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Centre Hospitalier Intercommunal Creteil

Créteil, , France

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Institut Cœur Poumon, CHU de Lille

Lille, , France

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Hopital Arnaud de Villeneuve

Montpellier, , France

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Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur

Nice, , France

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Hopital Cochin

Paris, , France

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Hopital Robert Debre

Paris, , France

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Centre Hospitalier Lyon Sud

Pierre-Bénite, , France

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Centre de Perharidy

Roscoff, , France

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CHU de Rouen - Hopital Charles Nicolle

Rouen, , France

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Hôpital de Hautepierre, AX5

Strasbourg, , France

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Hopital Foch (Suresnes), Hopital Foch, Adultes

Suresnes, , France

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Hopital Bretonneau

Tours, , France

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Charite Paediatric Pulmonology Department

Berlin, , Germany

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Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital

Erlangen, , Germany

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Kinderklinik III, Abt. fur Pneumologie

Essen, , Germany

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Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen

Essen, , Germany

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Johann Wolfgang Goethe University

Frankfurt, , Germany

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Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin

Giessen, , Germany

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Hannover Medical School

Hanover, , Germany

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Medizinische Hochschule Hannover

Hanover, , Germany

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Mukoviszidose-Zentrum am Universitätsklinikum Jena, Klinik für Kinder- und Jugendmedizin

Jena, , Germany

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Johannes Gutenberg-Universitaet

Mainz, , Germany

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Pneumologisches Studienzentrum Muenchen-West

München, , Germany

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Klinikum Westbrandenburg (CF)

Potsdam, , Germany

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Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF)

Ulm, , Germany

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Universitätsklinikum Würzburg

Würzburg, , Germany

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General Hospital of Thessaloniki "Papanikolaou"

Thessaloniki, , Greece

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General Hospital of Thessaloniki 'Hippokratio', 3rd Pediatrics Department of Aristotle University of Thessaloniki

Thessaloniki, , Greece

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National Koranyi Institute for TBC and Pulmonology

Budapest, , Hungary

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Pulmonology Institute Torokbalint

Törökbálint, , Hungary

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Cork University Hospital

Cork, , Ireland

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Children's Health Ireland at Crumlin

Dublin, , Ireland

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Children's Health Ireland at Temple Street

Dublin, , Ireland

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St. Vincent's University Hospital

Dublin, , Ireland

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University Hospital Limerick (Pediatrics)

Limerick, , Ireland

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Hadassah Medical Organization

Jerusalem, , Israel

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Schneider Children's Medical Center of Israel

Petach Tikvah, , Israel

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Sheba Medical Center - The Edmond and Lili Safra Children's Hospital

Tel Litwinsky, , Israel

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Azienda Ospedaliero Universitaria Ospedale Riuniti

Ancona, , Italy

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Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer

Florence, , Italy

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IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico

Genova, , Italy

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Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena

Milan, , Italy

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Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, , Italy

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Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica

Orbassano, , Italy

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Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo

Potenza, , Italy

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Azienda Ospedaliera di Verona - Ospedale Civile Maggiore

Verona, , Italy

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Academisch Medisch Centrum (Academic Medical Centre)

Amsterdam, , Netherlands

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UMC St. Radboud

Nijmegen, , Netherlands

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Erasmus Medical Center

Rotterdam, , Netherlands

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HagaZiekenhuis van den Haag

The Hague, , Netherlands

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University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis

Utrecht, , Netherlands

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Canterbury District Health Board

Christchurch, , New Zealand

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Starship Children's Hospital

Grafton, , New Zealand

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Waikato Hospital

Hamilton, , New Zealand

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Greenlane Clinical Centre

One Tree Hill, , New Zealand

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Haukeland Universitetssjukehus (CF)

Bergen, , Norway

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Oslo University Hospital, Department of Paediatric Medicine

Oslo, , Norway

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Pediatric Hospital Polanki named of Maciej Płażyński

Gdansk, , Poland

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Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY

Łomianki, , Poland

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Hospital de Santa Maria

Lisbon, , Portugal

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CHP - Hospital de Santo Antonio

Porto, , Portugal

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Hospital Sao Joao

Porto, , Portugal

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Hospital Saint Joan de Deu

Barcelona, , Spain

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Hospital Universitari Vall d'Hebron

Barcelona, , Spain

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Hospital Universitario 12 de Octubre

Madrid, , Spain

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Hospital Universitario Ramon y Cajal

Madrid, , Spain

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Hospital Virgen de la Arrixaca

Murcia, , Spain

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Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari

Sabadell, , Spain

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Hospital Universitario Virgen del Rocio

Seville, , Spain

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Hospital Universitario y Politecnico La Fe

Valencia, , Spain

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Sahlgrenska Universitetssjukhuset

Gothenburg, , Sweden

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Lund University Skanes Universitetssjukhus

Lund, , Sweden

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Karolinska Universitetssjukhuset, Huddinge

Stockholm, , Sweden

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Kinderspital Zuerich

Zurich, , Switzerland

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Birmingham Heartlands Hospital

Birmingham, , United Kingdom

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University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital

Bristol, , United Kingdom

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Royal Papworth Hospital NHS Foundation Trust

Cambridge, , United Kingdom

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Western General Hospital

Edinburgh, , United Kingdom

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Royal Devon University Healthcare NHS Foundation Trust

Exeter, , United Kingdom

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Clinical Research Facility, Queen Elizabeth University Hospital

Glasgow, , United Kingdom

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Leeds General Infirmary

Leeds, , United Kingdom

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St. James University Hospital

Leeds, , United Kingdom

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Liverpool Heart and Chest Hospital

Liverpool, , United Kingdom

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King's College Hospital

London, , United Kingdom

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Royal Brompton Hospital

London, , United Kingdom

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St. Bartholomew's Hospital

London, , United Kingdom

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Royal Manchester Children's Hospital

Manchester, , United Kingdom

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Wythenshawe Hospital

Manchester, , United Kingdom

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Clinical Research Facility

Newcastle upon Tyne, , United Kingdom

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All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough

Penarth, , United Kingdom

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Southampton General Hospital

Southampton, , United Kingdom

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Countries

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United States Australia Austria Belgium Canada Czechia Denmark France Germany Greece Hungary Ireland Israel Italy Netherlands New Zealand Norway Poland Portugal Spain Sweden Switzerland United Kingdom

Other Identifiers

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2021-000713-17

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2024-514173-22-00

Identifier Type: OTHER

Identifier Source: secondary_id

VX20-121-104

Identifier Type: -

Identifier Source: org_study_id

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